Department of Surgery, Division of Surgical Oncology, University of California, San Diego.
Department of Surgery, University of California, Los Angeles.
JAMA Netw Open. 2020 Sep 1;3(9):e2013565. doi: 10.1001/jamanetworkopen.2020.13565.
Gastrointestinal stromal tumor (GIST) is frequently driven by oncogenic KIT variations. Imatinib targeting of KIT marked a new era in GIST treatment and ushered in precision oncological treatment for all solid malignant neoplasms. However, studies on the molecular biological traits of GIST have found that tumors respond differentially to imatinib dosage based on the KIT exon with variation. Despite this knowledge, few patients undergo genetic testing at diagnosis, and empirical imatinib therapy remains routine. Barriers to genetic profiling include concerns about the cost and utility of testing.
To determine whether targeted gene testing (TGT) is a cost-effective diagnostic for patients with metastatic GIST from the US payer perspective.
DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation developed a Markov model to compare the cost-effectiveness of TGT and tailored first-line therapy compared with empirical imatinib therapy among patients with a new diagnosis of metastatic GIST. The main health outcome, quality-adjusted life years (QALYs), and costs were obtained from the literature, and transitional probabilities were modeled from disease progression and survival estimates from randomized clinical trials of patients with metastatic GIST. Data analyses were conducted October 2019 to January 2020.
TGT and tailored first-line therapy.
The primary outcome was QALYs and cost. Cost-effectiveness was defined using an incremental cost-effectiveness ratio, with an incremental cost-effectiveness ratio less than $100 000/QALY considered cost-effective. One-way and probabilistic sensitivity analyses were conducted to assess model stability.
Therapy directed by TGT was associated with an increase of 0.10 QALYs at a cost of $9513 compared with the empirical imatinib approach, leading to an incremental cost-effectiveness ratio of $92 100. These findings were sensitive to the costs of TGT, drugs, and health utility model inputs. Therapy directed by TGT remained cost-effective for genetic testing costs up to $3730. Probabilistic sensitivity analysis found that TGT-directed therapy was considered cost-effective 70% of the time.
These findings suggest that using genetic testing to match treatment of KIT variations to imatinib dosing is a cost-effective approach compared with empirical imatinib.
胃肠道间质瘤（GIST）常由致癌性 KIT 变异驱动。针对 KIT 的伊马替尼靶向治疗标志着 GIST 治疗的新时代，并为所有实体恶性肿瘤带来了精准的肿瘤治疗。然而，对 GIST 的分子生物学特征的研究发现，肿瘤对基于变异的 KIT 外显子的伊马替尼剂量反应不同。尽管有了这些认识，但很少有患者在诊断时进行基因检测，经验性伊马替尼治疗仍然是常规治疗。基因分析的障碍包括对检测成本和实用性的担忧。
从美国支付者的角度确定针对转移性 GIST 患者的靶向基因检测（TGT）是否具有成本效益。
设计、设置和参与者：这项经济评估建立了一个马尔可夫模型，以比较 TGT 和靶向一线治疗与经验性伊马替尼治疗在新诊断为转移性 GIST 的患者中的成本效益。主要健康结果（质量调整生命年[QALY]）和成本来自文献，转移概率来自转移性 GIST 患者随机临床试验的疾病进展和生存估计。数据分析于 2019 年 10 月至 2020 年 1 月进行。
TGT 和靶向一线治疗。
主要结果是 QALY 和成本。成本效益定义为增量成本效益比，增量成本效益比低于 100000 美元/QALY 被认为具有成本效益。进行了单因素和概率敏感性分析，以评估模型稳定性。
与经验性伊马替尼方法相比，TGT 指导的治疗与 QALY 增加 0.10 相关，成本增加 9513 美元，导致增量成本效益比为 92100 美元。这些发现对 TGT、药物和健康效用模型输入的成本敏感。TGT 指导的治疗在基因检测成本高达 3730 美元时仍然具有成本效益。概率敏感性分析发现，TGT 指导的治疗在 70%的时间内被认为具有成本效益。
这些发现表明，与经验性伊马替尼相比，使用基因检测来匹配 KIT 变异的治疗与伊马替尼剂量是一种具有成本效益的方法。
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